Phase
Condition
Eye Disorders/infections
Retinitis Pigmentosa
Posterior Uveitis
Treatment
rAAV2tYF-GRK1-hRPGRco
Control
Clinical Study ID
Ages 12-50 Male
Study Summary
Eligibility Criteria
Inclusion
General Inclusion Criteria:
Provide written informed consent or assent (per local regulation), prior to theconduct of any study-related procedure. Participants who provide assent must have aparent, guardian, or legal representative provide written informed consent.
Be between 12 and 50 years of age (inclusive) at the time of informed consent andassent (as applicable).
Be male (XY chromosome) and have at least one documented pathogenic or likelypathogenic variant in the RPGR gene.
Have a clinical diagnosis of XLRP.
Be able and willing, as assessed by the Investigator, to follow study instructions,complete study assessments, comply with the protocol, and attend study visits forthe duration of the study. Ocular Inclusion Criteria (Study Eye):
Have a BCVA ≤ 78 letters (approximately Snellen, 20/32) and ≥ 34 letters (approximately Snellen, 20/200)
Have a LLVA ≤64 letters (approximately Snellen 20/50) in the study eye
Be able to perform all tests of visual and retinal function and structure in botheyes based on the participant's reliability, and fixation, in the study eye per theInvestigator's discretion.
Have detectable baseline mean macular sensitivity .
Have a detectable sub-foveal ellipsoid zone (EZ) line as assessed by SD-OCT in thestudy eye and confirmed by the CRC.
If study eye will be at the discretion of the Investigator and/or Surgeon.
Exclusion
General Exclusion Criteria:
Have other known disease-causing mutations documented in the participant's medicalhistory or identified through a retinal dystrophy gene panel, that in the opinion ofthe Investigator would interfere with the potential therapeutic effect of the studyagent or the quality of the assessments.
For participants with herpes simplex virus (HSV):
Have history of oral or genital herpes and unable and/or unwilling to utilizeprophylactic antiviral medication.
Have a history of ocular herpes.
Have active oral or genital herpes or are currently receiving treatment for HSVinfection.
Have known sensitivity or allergy to systemic corticosteroids or otherimmunosuppressive medications.
Have used anti-coagulant agents that may alter coagulation (e.g., warfarin, heparin,apixaban, or high dose docosahexaenoic acid [DHA; fish oil]) within 7 days prior tostudy treatment administration (ibuprofen, aspirin, or similar are acceptable).
Have used systemic corticosteroids or other immunosuppressive medications within 3months prior to screening and/or intend to use during screening. Corticosteroidsused on an as-needed basis administered by insufflation, inhalation or localadministration to the skin and mucosa such as Symbicort (budesonide/formoterol),Flonase (fluticasone propionate), and skin creams and ointments containingcorticosteroids shall not be exclusionary.
If sexually active or planning to become sexually active, are unwilling to usebarrier contraception for 3 months following treatment administration.
Are currently participating or recently participated in any other research
Have previously received any AAV gene therapy product, stem cell therapy, cell-basedtherapy, or similar biologics.
Have significant media opacity impacting evaluation of the retina or vitreous.administration.
Had intraocular surgery within 90 days of study treatment administration.
Have any active ocular/intraocular infection or inflammation (e.g., severeblepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis,idiopathic or autoimmune associated uveitis, or herpetic lesions).
Have a history of corticosteroid-induced raised IOP of >25 mmHg followingcorticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
Have any artificial retinal implant or prosthesis.
Have absence of clear ocular media and/or inadequate pupil dilation to facilitategood quality SD-OCT images.
Have any history of rhegmatogenous retinal detachment.
Have myopia (spherical equivalent) exceeding -10 diopters (or axial length of >30 mmif the Principal Investigator [PI] deems it appropriate to measure) or presence ofpathologic myopia in the study eye.
Have passed the Low Contrast Ora-VNC mobility course at ≤0.35 lux light level ineither eye or binocularly at any screening visit.
Study Design
Study Description
Connect with a study center
Sydney Eye Hospital
Sydney, New South Wales 2000
AustraliaSite Not Available
Royal Victorian Eye & Ear Hospital
East Melbourne, Victoria 3002
AustraliaSite Not Available
Hadassah Medical Center
Jerusalem,
IsraelSite Not Available
Radboud University Medical Center
Nijmegen,
NetherlandsSite Not Available
Rotterdam Ophthalmic Institute
Rotterdam,
NetherlandsSite Not Available
Moorfields Eye Hospital
London, EC1V 2PD
United KingdomSite Not Available
The Retina Clinic London, Institute of Ophthalmology, University College London
London, W1G7LB
United KingdomSite Not Available
Oxford Eye Hospital
Oxford, OX39DU
United KingdomActive - Recruiting
Retina Macula Institute of Arizona
Scottsdale, Arizona 85255
United StatesSite Not Available
University of San Diego
San Diego, California 92110
United StatesSite Not Available
Colorado Retina Associates
Denver, Colorado 80230
United StatesSite Not Available
University of Florida
Gainesville, Florida 32611
United StatesSite Not Available
University of Florida Health Jacksonville, Department of Ophthalmology
Jacksonville, Florida 32209
United StatesActive - Recruiting
Bascom Palmer Eye Institute- University of Miami
Miami, Florida 33136
United StatesActive - Recruiting
Midwest Eye Institute (Retina Partners Midwest)
Carmel, Indiana 46290
United StatesSite Not Available
Wilmer Eye Institute at Johns Hopkins
Baltimore, Maryland 21287
United StatesSite Not Available
Boston Children' Hospital
Boston, Massachusetts 02115
United StatesSite Not Available
Ophthalmic Consultants of Boston
Boston, Massachusetts 02114
United StatesSite Not Available
Mayo Clinic
Rochester, Minnesota 55905
United StatesActive - Recruiting
Duke Eye Center
Durham, North Carolina 27705
United StatesSite Not Available
Cincinnati Eye Institute
Cincinnati, Ohio 45242
United StatesActive - Recruiting
Cleveland Clinic - Cole Eye Institute
Cleveland, Ohio 44195
United StatesSite Not Available
Cole Eye Institute - Cleveland Clinic
Cleveland, Ohio 44195
United StatesActive - Recruiting
Casey Eye Institute, OHSU
Portland, Oregon 97239
United StatesActive - Recruiting
Casey Eye Institute, Oregon Health and Sciences University
Portland, Oregon 97239
United StatesSite Not Available
Mid Atlantic Retina
Philadelphia, Pennsylvania 19107
United StatesActive - Recruiting
University of Pittsburgh
Pittsburgh, Pennsylvania 15219
United StatesActive - Recruiting
Retina Consultants of Texas
Bellaire, Texas 77401
United StatesActive - Recruiting
Retina Foundation of the Southwest
Dallas, Texas 75231
United StatesActive - Recruiting
Baylor Eye Institute
Houston, Texas 77030
United StatesActive - Recruiting
Retina Consultants of San Antonio Texas
San Antonio, Texas 78240
United StatesActive - Recruiting
Retina Consultants of Texas- San Antonio
San Antonio, Texas 78240
United StatesActive - Recruiting
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